Direct gene transfer into mouse diaphragm

Abstract

Direct gene transfer into skeletal muscle is a potential therapeutic strategy for inherited primary myopathies such as Duchenne muscular dystrophy (DMD). In order to affect the life‐expectancy of these patients, it will be necessary to carry out gene therapy on the diaphragm. To this end, we report efficient introduction of pure recombinant plasmid DNA into the mouse diaphragm, without causing significant damage. Application of this approach to the diaphragm of the mdx mouse will provide information on the potential usefulness of gene therapy for the treatment of DMD patients.